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Clinical Trials in China / NCT07054632
Active, not recruiting Phase 3

Efficacy and Safety of LX101 for Inherited Retinal Dystrophy Associated With RPE65 Mutations

NCT07054632 · tracked via the Priya Life Science China tracker
Sponsor
Innostellar Biotherapeutics Co.,Ltd
Phase
Phase 3
Started
2023-09-13
Last updated
2026-03-05

Condition(s) studied

Inherited Retinal Dystrophy Associated With RPE65 Mutations

Investigational drug(s) / intervention(s)

LX101

LX101: Subretinal administration of LX101 to the study eye

Study summary

The purpose of the study is to evaluate the efficacy and safety of LX101 in subjects with biallelic RPE65 mutation-associated inherited retinal dystrophy. This is an open-label, multicenter, randomized controlled Phase III clinical trial. Subjects were randomly assigned in a 1:1 ratio to either the intervention group or the control group. Subjects in the intervention group received subretinal injection of LX101, while those in the control group received no treatment.

Eligibility

Sex
ALL
Min age
6 Years
Max age
Healthy volunteers
No
Inclusion Criteria: * Subject and/or their guardian signing a written informed consent. Diagnosed with biallelic RPE65 mutation-associated inherited retinal dystrophy. Subjects are 6 years of age or older. Visual acuity of ≤ 20/63 or visual field less than 20 degrees in the eye to be injected. Exclusion Criteria: * Prior gene therapy for IRD and other hereditary eye diseases. Pre-existing eye conditions that would interfere with interpretation of study endpoints. Active intraocular or periocular infections in the study eye. Lacking of sufficient surviving retinal cells. Prior ocular surgery within six months. Complicating systemic diseases or clinically significant abnormal baseline laboratory values. Pre-existing systemic diseases that should not discontinue the use of any retinal toxic compounds. Complicating systemic diseases or clinically significant abnormal baseline laboratory values.

Primary outcome measure(s)

Trial sites (4)

FacilityCityRegionStatus
Zhongshan Ophthalmic Center, Sun Yat sen University Guangzhou Guangdong
Southwest Hospital of AMU Chongqing China
Shanghai General Hospital Shanghai China
Tianjin Medical University Ophthalmology Hospital Tianjin China

More Innostellar Biotherapeutics Co.,Ltd trials in China

Other trials for the same condition

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT07054632 on ClinicalTrials.gov ↗ ← All trials in China